Proceedings of the Japan Academy, Series B
Online ISSN : 1349-2896
Print ISSN : 0386-2208
ISSN-L : 0386-2208
Reviews
Progress in muscular dystrophy research with special emphasis on gene therapy
Hideo SUGITAShin’ichi TAKEDA
Author information
JOURNAL FREE ACCESS

2010 Volume 86 Issue 7 Pages 748-756

Details
Abstract

Duchenne muscular dystrophy (DMD) is an X-linked, progressive muscle-wasting disease caused by mutations in the DMD gene. Since the disease was described by physicians in the 19th century, information about the subject has been accumulated. One author (Sugita) was one of the coworkers who first reported that the serum creatine kinase (CK) level is elevated in progressive muscular dystrophy patients. Even 50 years after that first report, an elevated serum CK level is still the most useful marker in the diagnosis of DMD, a sensitive index of the state of skeletal muscle, and useful to evaluate therapeutic effects. In the latter half of this article, we describe recent progress in the therapy of DMD, with an emphasis on gene therapies, particularly exon skipping.

(Communicated by Kunihiko SUZUKI, M.J.A.)

Content from these authors
© 2010 The Japan Academy
Previous article Next article
feedback
Top